ArticleFrontiers in oncology2021
Phase 2 Study of Pomalidomide (CC-4047) Monotherapy for Children and Young Adults With Recurrent or Progressive Primary Brain Tumors.
Article in Frontiers in oncology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03257631 (A PHASE 2 CLINICAL STUDY OF POMALIDOMIDE), which is not on this map. Cited by 11 papers, 3 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A phase 2 clinical study of pomalidomide (cc-4047) monotherapy for children and young adults with recurrent or progressive primary brain tumors
Who cites it
11 citing papers in PubMed, 3 syntheses or guidelines pooled it, 11 citations in OpenAlex.
- Outcomes of immunotherapy in medulloblastoma: a systematic review.The oncologist · 2026Pooled it
- Evaluation of two-stage designs of Phase 2 single-arm trials in glioblastoma: a systematic review.BMC medical research methodology · 2022Pooled it
- A systematic review of ongoing clinical trials in optic pathway gliomas.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2020Pooled it
- H3 K27M-altered glioma and diffuse intrinsic pontine glioma: Semi-systematic review of treatment landscape and future directions.Neuro-oncology · 2024Review
- Indoximod-based chemo-immunotherapy for pediatric brain tumors: A first-in-children phase I trial.Neuro-oncology · 2024Article
- Review
- Emerging Therapeutic Strategies for Diffuse Intrinsic Pontine Glioma: A Systematic Review.Healthcare (Basel, Switzerland) · 2023Review
- Epigenetics and immune cells in medulloblastoma.Frontiers in genetics · 2023Review
- Immunotherapy for the treatment of pediatric brain tumors: a narrative review.Translational pediatrics · 2022Review
- Recurrent or progressive pediatric brain tumors: population pharmacokinetics and exposure-response analysis of pomalidomide.Pediatric research · 2021Article
- Neuroinflammation as a Factor of Neurodegenerative Disease: Thalidomide Analogs as Treatments.Frontiers in cell and developmental biology · 2019Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
16 authors at 9 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionTreatment of recurrent primary pediatric brain tumors remains a major challenge, with most children succumbing to their disease. We conducted a prospective phase 2 study investigating the safety and efficacy of pomalidomide (POM) in children and young adults with recurrent and progressive primary brain tumors.
methodsPatients with recurrent and progressive high-grade glioma (HGG), diffuse intrinsic pontine glioma (DIPG), ependymoma, or medulloblastoma received POM 2.6 mg/m
results46 patients were evaluable for response (HGG, n = 19; DIPG, ependymoma, and medulloblastoma, n = 9 each). Two patients with HGG achieved OR or LTSD (10.5% [95% CI, 1.3%-33.1%]; 1 partial response and 1 LTSD) and 1 patient with ependymoma had LTSD (11.1% [95% CI, 0.3%-48.2%]). There were no ORs or LTSD in the DIPG or medulloblastoma cohorts. The median PFS for patients with HGG, DIPG, ependymoma, and medulloblastoma was 7.86, 11.29, 8.43, and 8.43 weeks, respectively. Median OS was 5.06, 3.78, 12.02, and 11.60 months, respectively. Neutropenia was the most common grade 3/4 adverse event.
conclusionsTreatment with POM monotherapy did not meet the primary measure of success in any cohort. Future studies are needed to evaluate if POM would show efficacy in tumors with specific molecular signatures or in combination with other anticancer agents. CLINICAL
trial registrationClinicalTrials.gov, identifier NCT03257631; EudraCT, identifier 2016-002903-25.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.